CA3164189A1 - Adeno associated virus vectors for the treatment of hunter disease - Google Patents
Adeno associated virus vectors for the treatment of hunter diseaseInfo
- Publication number
- CA3164189A1 CA3164189A1 CA3164189A CA3164189A CA3164189A1 CA 3164189 A1 CA3164189 A1 CA 3164189A1 CA 3164189 A CA3164189 A CA 3164189A CA 3164189 A CA3164189 A CA 3164189A CA 3164189 A1 CA3164189 A1 CA 3164189A1
- Authority
- CA
- Canada
- Prior art keywords
- raav
- vector
- subject
- activity
- human
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
- C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
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- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
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| US201962945920P | 2019-12-10 | 2019-12-10 | |
| US62/945,920 | 2019-12-10 | ||
| PCT/US2020/063887 WO2021119053A1 (en) | 2019-12-10 | 2020-12-09 | Adeno associated virus vectors for the treatment of hunter disease |
Publications (1)
| Publication Number | Publication Date |
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| CA3164189A1 true CA3164189A1 (en) | 2021-06-17 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
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| CA3164189A Pending CA3164189A1 (en) | 2019-12-10 | 2020-12-09 | Adeno associated virus vectors for the treatment of hunter disease |
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| Country | Link |
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| US (1) | US11981912B2 (es) |
| EP (1) | EP4072595A1 (es) |
| JP (1) | JP2023505851A (es) |
| KR (1) | KR20220112283A (es) |
| AR (1) | AR120709A1 (es) |
| AU (1) | AU2020401116A1 (es) |
| BR (1) | BR112022011332A2 (es) |
| CA (1) | CA3164189A1 (es) |
| CO (1) | CO2022009604A2 (es) |
| IL (1) | IL293684A (es) |
| MX (1) | MX2022007135A (es) |
| TW (1) | TW202136515A (es) |
| WO (1) | WO2021119053A1 (es) |
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| KR20220164743A (ko) * | 2020-04-06 | 2022-12-13 | 호몰로지 메디슨, 인크. | Ids 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 |
| WO2023187728A1 (en) * | 2022-04-01 | 2023-10-05 | Takeda Pharmaceutical Company Limited | Gene therapy for diseases with cns manifestations |
Family Cites Families (35)
| Publication number | Priority date | Publication date | Assignee | Title |
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| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| EP1007637B1 (en) | 1997-04-14 | 2004-06-30 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| CA2348382C (en) | 1998-11-10 | 2013-09-17 | The University Of North Carolina At Chapel Hill | Chimeric parvovirus vectors and methods of making and administering the same |
| WO2001091803A2 (en) | 2000-06-01 | 2001-12-06 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| US7985553B2 (en) * | 2001-10-29 | 2011-07-26 | Nathaniel Heintz | Method for isolating cell type-specific mRNAs |
| PL222683B1 (pl) | 2001-11-13 | 2016-08-31 | Univ Pennsylvania | Rekombinowane wirusy stowarzyszone z adenowirusem (AAV), sposoby ich wytwarzania, pakująca komórka gospodarza, kompozycja zawierająca wirus, zastosowanie wirusa, wyizolowane wirusy AAV, białka,sztuczne białka kapsydu, cząsteczki, komórki gospodarza, sposoby dostarczania transgenu, sposób identyfikacji serotypu, zestaw diagnostyczny, sposób izolacji nowych wirusów, rekombinowana komórka |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
| CN102199626B (zh) | 2003-09-30 | 2015-06-24 | 宾夕法尼亚大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| DK2359867T3 (en) | 2005-04-07 | 2015-01-05 | Univ Pennsylvania | A method for increasing an AAV vector function |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| PL3252161T3 (pl) | 2007-06-06 | 2022-03-07 | Genzyme Corporation | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| JP6023073B2 (ja) | 2010-12-22 | 2016-11-09 | フォンダッツィオーネ・テレソン | ムコ多糖症におけるｃｎｓ病変を処置するための治療戦略 |
| KR20130131461A (ko) | 2011-03-07 | 2013-12-03 | 폰다지오네 텔레톤 | Tfeb 변종 및 그의 용도 |
| US8609088B2 (en) | 2011-05-10 | 2013-12-17 | Regents Of The University Of Minnesota | Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases |
| WO2012158757A1 (en) | 2011-05-16 | 2012-11-22 | The Trustees Of The University Of Pennsylvania | Proviral plasmids for production of recombinant adeno-associated virus |
| WO2013156625A1 (en) | 2012-04-19 | 2013-10-24 | Fondazione Telethon | Tfeb gene therapy of alpha-1-antitrypsin deficiency |
| BR112015008673A2 (pt) | 2012-10-17 | 2017-09-26 | Fond Telethon | composto, composição farmacêutica e método |
| BR112015028605A8 (pt) | 2013-05-15 | 2019-12-24 | Univ Minnesota | uso de uma composição e uso de um imunossupressor e uma composição |
| GB201308917D0 (en) | 2013-05-17 | 2013-07-03 | Renishaw Plc | Delivery |
| US20160243260A1 (en) | 2013-10-24 | 2016-08-25 | Uniqure Ip B.V. | Treatment of neurological diseases using adeno-associated virus (AAV) comprising AAV-5 capsid proteins |
| ES2901766T3 (es) | 2015-05-15 | 2022-03-23 | Regenxbio Inc | Virus adenoasociado para el suministro terapéutico al sistema nervioso central |
| EP3101125A1 (en) * | 2015-06-05 | 2016-12-07 | Laboratorios Del Dr. Esteve, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
| AU2017250298B2 (en) | 2016-04-15 | 2024-10-03 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| KR20190086503A (ko) * | 2016-11-15 | 2019-07-22 | 리젠츠 오브 더 유니버시티 오브 미네소타 | Mpsi 및 mpsii에서의 신경계 기능 및 다른 신경계 장애를 개선하는 방법 |
| AU2018335752A1 (en) * | 2017-09-22 | 2020-03-12 | Christian HINDERER | Gene therapy for treating Mucopolysaccharidosis type ii |
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| BR112022011332A2 (pt) | 2022-08-23 |
| KR20220112283A (ko) | 2022-08-10 |
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| US20210332383A1 (en) | 2021-10-28 |
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| MX2022007135A (es) | 2022-09-19 |
| US11981912B2 (en) | 2024-05-14 |
| IL293684A (en) | 2022-08-01 |
| WO2021119053A1 (en) | 2021-06-17 |
| AU2020401116A1 (en) | 2022-07-21 |
| TW202136515A (zh) | 2021-10-01 |
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